Release date: 2017-03-24
According to the latest report, the cause of blindness may be that the material injected into the patient's eye destroys the enzyme system of the eye. Or when stem cells are injected into the eye, they are transformed into myofibroblasts and cause scarring in the eye. For a time, it has caused a global debate. What is the future of ophthalmology?
Last Wednesday, a female patient received so-called adipose tissue-derived stem cell therapy, leading to complete blindness. According to the latest report, the cause of blindness may be that the material injected into the patient's eye destroys the enzyme system of the eye. Or when stem cells are injected into the eye, they are transformed into myofibroblasts and cause scarring in the eye. For a time, it has caused a global debate. What is the future of ophthalmology?
Dr. Thomas Albini, an ophthalmologist at the University of Miami, explained that the plan used in this medical accident did not prove to be effective or safe in the human body. Therefore, a large part of the reason is attributed to treatment supervision. When it is possible to cure patients, we need to provide safe and legal treatment.
However, in order for the majority of patients to see the light as soon as possible, scientists have not slowed down the search for new treatments for eye diseases.
On March 20th, NightstaRx announced the start of Phase I clinical trial of gene therapy for X-linked retinitis pigmentosa, the second AAV candidate tested by patients at NightstaRx.
NightstaRx is a biotechnology company that develops gene therapy for patients with hereditary retinal dystrophy. It is a biotechnology company in clinical research and is headquartered in the UK.
As early as January 2014, the company collaborated with the University of Oxford to develop a gene therapy drug, AAV2-REP1, for patients with choroidal disease.
Robert MacLaren
The drug was developed by Professor Robert MacLaren of the Nuffield Eye Laboratory at the University of Oxford to treat gene therapy for hereditary progressive blindness without choroidal disease. The treatment of clinical trials has shown promising results, and the results were published in the Lancet. This method uses a recombinant adeno-associated virus (AAV) viral vector to deliver a Rab-escort protein 1 (REP-1)-based (AAV2-REP1) wild-type copy to retinal cells in choroid-free patients. The study is currently underway and the next six patients will receive higher doses. The company will soon announce its Phase III research plan.
Retinitis Pigmentosa (RP) is a hereditary, progressive, dystrophic, chronic retinal pigmented degeneration, most of which is caused by both eyes. Once the retina produces lesions, vision will be degraded due to the sharp decrease in the number of rods in the retina (visual cells for dark vision) and cones (one of specialized retinal receptors).
RP is highly genetically heterogeneous with a variety of different phenotypes and inheritance patterns. Among them, X-linked retinitis pigmentosa (XLRP) is a sexually associated recessive genetic disease, which inevitably leads to blindness in adult males. The affected people are blind at an average age of 45 years. XLRP constitutes a group of genetic diseases that affect the retinal response to light, especially caused by abnormalities in the photoreceptors at the back of the eye. In more than two-thirds of cases, the disease is caused by mutations in the retinitis pigment GTPase modulator (RPGR) gene. Common early symptoms include difficulty seeing young men and gradual loss of vision and vision at night. There is currently no effective treatment.
For the company's second AAV candidate, I have begun recruiting and treating subjects for Phase I / II clinical trials, the first clinical trial of the global XLRP type, and only 18 have been used from licensing to clinical trials. The time of the month.
Just last Thursday, a 29-year-old man became the first person in the world to undergo surgery at the Oxford Eye Hospital and is now recovering.
Robert MacLaren, professor of ophthalmology and principal researcher at Oxford University, said: "The treatment is going well, but we need to wait a few years to know if the patient has stopped retinal degeneration. The effect of the disease of retinitis pigmentosa on the family is devastating. We spent many years researching how to develop this gene therapy. Changing the genetic code is always very cautious, but the new sequences we have used have proven to be very effective in our laboratory research. All life genetic codes on Earth are G, T, A and C consisting of four bases, but in the RPGR gene, only A and G are included. This makes the RPGR gene very unstable and prone to mutation, making it the main cause of blindness in patients with retinitis pigmentosa. the reason."
The gene therapy approach developed by NightstaRx is the use of a vector of adeno-associated virus (AAV) to deliver a codon-optimized copy of the retinitis pigmentosa GTPase modulator (RPGR) gene into eye cells for the first time. For clinical testing.
Aniz Girach
Dr. Aniz Girach, Chief Medical Officer of NightstaRx commented: "The current trial is a multi-center open-label, dose-increasing study that recruits at least 24 male patients, such as Oxford and Manchester, for centers of excellence in ophthalmology. Each patient will A single subretinal injection of the AAV-XLRPGR gene therapy. The primary objective of the study was to evaluate the safety and tolerability of AAV-XLRPGR within 12 months.
Robert MacLaren commented: "X-linked retinitis pigmentosa (XLRP) is a devastating disease that causes early onset of blindness in men. Many individuals are legally blind in their teens and there is currently no viable treatment. Based on previous The findings in the preclinical in vivo disease model show a significant response to photoreceptors, which we believe has the potential to restore or maintain patient vision. Unique codon optimization strategies overcome the inherent instability of RPGR problem.
David Fellows
David Fellows, CEO of NightstaRx, said that we are pleased to report on the company's second lead in hereditary retinal malnutrition. If successful, this gene therapy will benefit more patients with eye diseases. "
In the current era, gene therapy technology is changing with each passing day. The past terminal illness like hereditary diseases can be improved and restored by gene therapy. Gene therapy technology is the most feasible technology for treating hereditary diseases. However, it is still necessary for NightstaRx to carefully consider and study whether the determination of the decisive lesion site of the hereditary disease and the change of its site will cause other types of diseases. We hope that more companies like NightstaRx's gene-based drugs are committed to becoming bright messengers, lighting up the world for these patients with eye diseases.
Source: Medical Maike
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